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Gene Therapy Clinical Trials for Sanfilippo A and B Coming in 2014

I have some questions about the Gene Therapy that has been posted recently by Team Sanfilippo.

Here is a snippet of what has been posted on the Team Sanfilippo website:

“A consortium of family foundations from around the world have come together to fund clinical trials in 2014 for Sanfilippo A and B using gene therapy. The trials will be conducted at Nationwide Children’s Hospital in Columbus, Ohio.

The two lead products, ABX-A and ABX-B, uniquely deliver the therapeutic product to the CNS with the aim of preventing and/or reversing the effects of the genetic errors that cause the disease. ABX-A and ABX-B induce cells in the CNS and digestive tract to produce the missing enzymes and help repair and prevent damage caused to the cells. Safety studies conducted in large animal models have demonstrated that delivery of ABX-A and ABX-B are well-tolerated with minimal side effects. Importantly, efficacy studies in animals with Sanfilippo syndrome have demonstrated unprecedented therapeutic benefit months after treatment. A single dose of ABX-A or ABX-B significantly restored normal cell and organ function and increased the lifespan of animals with SF over 100% a year after treatment compared to untreated control animals. Similarly, animals treated with ABX-A and ABX-B demonstrated corrections of cognitive defects that remained months after drug administration. These results are consistent with studies from several laboratories suggesting ABX-A and ABX-B treatment could potentially benefit patients with Sanfilippo Syndrome Type A and B, respectively.

ABX-A and ABX-B will be administered systemically for efficient drug delivery to the whole body and CNS – a significant advantage over current therapy options.”

Jake is not in the Natural History Study, so I am not up to date on the latest and greatest. Some questions that come to mind are:

1. Is this different than the Shire trial they are doing overseas? (I am guessing it is.)

2. What is the vehicle used to administer these products into the CNS?

3. Does anyone have any idea what will be involved with the trial, i.e. how many treatments?

Gene therapy is different from the IT trials. In the IT trials, they are injecting the enzyme directly into the spinal fluid to allow it to reach the brain. With gene therapy, they splice the missing or mutated gene into a certain kind of virus (with the bad parts taken out). The virus travels through the body and actually puts that gene into the cells, allowing the body to produce the enzyme on its own. Depending on the virus that they use, it may have to be repeated periodically, but it wouldn't be as frequent as ERT or IT enzyme. In theory, gene therapy could be a cure rather than a treatment. This is my simplistic explanation- my husband could lecture on it for days! His early research in grad school was related to gene therapy (not for MPS).

Good explanation Robin!
There is one other gene therapy trial that has already started in Paris involving 4 young Sanfilippo patients. There are no results yet, but this gene therapy of Dr. Fu's is far less invasive. that it will start punctually.

I've been asking around (ie. sending an email to Team Sanfilippo, filling out a contact form for the children's hospital directly) to see how to get more information on how to be considered for the trial. Do any of you have info on how to become a candidate for it? Who do I get in touch with?

Natural Hist Study

We have 2 children with Sanfilippo B, and I inquired about the natural hist study being carried out in Columbus Ohio. I received a call last night from Dr. Kevin Flanagan from Nationwide Children's Hospital. He explained that they used a virus as a vehicle.